Increased Lyso-Gb1 Levels in an Obese Splenectomized Gaucher Disease Type 1 Patient Treated with Eliglustat: Unacknowledged Poor Compliance or Underlying Factors

Increased Lyso-Gb1 Levels in an Obese Splenectomized Gaucher Disease Type 1 Patient Treated with Eliglustat: Unacknowledged Poor Compliance or Underlying Factors

Eliglustat (Cerdelga<sup>®</sup>) is a potent and specific inhibitor of the enzyme glucosylceramide synthase and serves as a substrate reduction therapy for adult patients with Gaucher disease type 1 (GD1). It prevents the accumulation of several lipids, including glucosylsphingosine (al...

Full description

Saved in:
Bibliographic Details
Main Authors: Evelina Maines, Roberto Franceschi, Giacomo Luppi, Giacomo Marchi, Giovanni Piccoli, Nicola Vitturi, Massimo Soffiati, Annalisa Campomori, Silvana Anna Maria Urru
Format: Article
Language:English
Published: MDPI AG 2025-06-01
Series:Metabolites
Subjects:
Gaucher disease
eliglustat
LysoGb1
splenectomy
obesity
case report
Online Access:https://www.mdpi.com/2218-1989/15/7/427
Tags: Add Tag
No Tags, Be the first to tag this record!

Internet

https://www.mdpi.com/2218-1989/15/7/427

Similar Items